cystic fibrosis transmembrane conductance regulator gene-lipid complex

**Cystic fibrosis transmembrane conductance regulator gene-lipid complex** is an experimental non-viral gene-replacement therapy for cystic fibrosis. It comprises plasmid DNA encoding functional human cystic fibrosis transmembrane conductance regulator delivered to airway epithelial cells using a cationic lipid carrier. The intended effect is transgene expression and restoration of CFTR-dependent chloride and bicarbonate transport, thereby addressing the underlying molecular defect rather than modulating residual mutant CFTR protein. The Genzyme-originated program reached Phase 1 development and was subsequently halted. ([synapse.patsnap.com](https://synapse.patsnap.com/drug/242ffd53f58340c49ff9ca5a97f61004))

Other names

CFTR gene-cationic lipid complexCFTR gene-cytofectin complexcystic fibrosis gene therapy